Central Nervous System Disorders
Investigators at University of Bonn publish new data on central nervous system disorders
Published in Gene Therapy Weekly, June 11th, 2009
"Inherited deficiencies of lysosomal hydrolases cause lysosomal storage diseases (LSDs) that are characterized by a progressive multisystemic pathology and premature death. Repeated intravenous injection of the active counterpart of the deficient enzyme, a treatment strategy called enzyme replacement therapy (ERT), evolved as a clinical option for several LSDs without central nervous system (CNS) involvement," researchers in Bonn, Germany report.
"To assess the efficacy of long-term ERT in metachromatic leukodystrophy (MLD), an LSD with prevailing nervous system disease, we treated immunotolerant arylsulfatase A (ASA) knockout mice with 52 doses of either 4 or 50 mg/kg...
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Source: Gene Therapy Weekly (2009-06-11)
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